Leaders in Complement January 2022 Forward-looking statements Statements in this presentation about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "wo ...

Financial Data and Key Metrics Changes - Total expenses increased by $396,000 quarter over quarter to $2,700,000 in Q1 2025 compared to $2,300,000 in Q1 2024 [15] - Research and development expenses rose by $289,000 to $1,500,000 in Q1 2025 from $1,200,000 in Q1 2024 [15] - Net loss was $2,700,000 in Q1 2025 compared to $2,300,000 in Q1 2024 [16] - Net cash used in operations was $1,600,000 for Q1 2025, comparable to $1,500,000 in Q1 2024 [17] Business Line Data and Key Metrics Changes - The CARDI Amp heart failure trial demonstrated safety and meaningful benefits for heart failure patients [6] - The CardioM Heart Failure II trial is actively enrolling patients at three clinical sites, with expectations for full enrollment over the next two years [7] - The Helix biotherapeutics delivery system is preparing for submission for approval, with potential value for therapeutic partners [9] Market Data and Key Metrics Changes - The electrophysiology market is valued at over $10 billion per year and is expanding to treat arrhythmias in the ventricles of the heart [12] - The company is focused on the Japanese market, with expectations for regulatory approval processes similar to the FDA [30] Company Strategy and Development Direction - The company aims to align with the FDA and Japan's PMDA to make CARDI Amp therapy available to physicians and patients [6] - Business development efforts are focused on partnerships that enhance shareholder value across all four platforms: CardiAmp, Cardiallo, Helix, and MorphDNA [10] - The company is open to partnerships in various indications not currently pursued, particularly in the allogeneic cell therapy space [11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the safety and efficacy of their therapies, emphasizing the importance of generating additional clinical evidence [40] - The company is optimistic about the potential for significant market opportunities in Japan, particularly with the CARDI Amp therapy [35] - Management noted that they are not significantly impacted by tariffs due to the domestic manufacturing of most components [49] Other Important Information - The company completed a small financing with minimal dilution to support upcoming milestones [14] - Management highlighted the importance of physician outreach and training for the introduction of new therapies into commercial channels [32] Q&A Session Summary Question: Discussion on business development maturity levels - Management indicated that while deal discussions can be lengthy, they have established products and ongoing discussions with large strategic partners [20] Question: Importance of Japan PMDA submission for CARDI Amp - Management confirmed that receiving permission to submit for approval in Japan is critical and aligns with their strategy for market entry [30] Question: Interaction between ongoing trials and FDA submission - Management clarified that they are continuously developing evidence while submitting for FDA approval, focusing on patient enrollment and data generation [40]

Core Insights - MeiraGTx Holdings plc is showcasing four poster presentations at the ASGCT 2025 Annual Meeting, highlighting advancements in gene and cell therapy technologies [1][19] Group 1: Poster Presentations - Poster 507 discusses an ultra-low dose localized CNS gene therapy targeting severe pediatric obesity, utilizing BDNF delivered via AAV to improve metabolic health and prevent severe obesity-related conditions [3][4][5] - Poster 893 presents novel CAG-based promoters that outperform traditional promoters in both mouse and human models, aiming to enhance gene therapy safety and efficacy by reducing required dosages [6][7][8][9][10] - Poster 897 explores the differential usage of transcription factor binding sites to enhance synthetic promoter activity, demonstrating significant improvements in gene expression through optimized designs [11][12][13][14] - Poster 1040 details the preclinical efficacy of an optimized AAV-hUPF1 gene therapy for ALS and Frontotemporal Dementia, showing promise in improving motor neuron survival and therapeutic efficacy [15][16][18] Group 2: Company Overview - MeiraGTx is a vertically integrated, clinical-stage genetic medicines company with a diverse pipeline, including four late-stage clinical programs targeting inherited and common diseases [19][20] - The company has developed a proprietary manufacturing platform and possesses comprehensive manufacturing capabilities, including GMP facilities for viral vector production [20] - MeiraGTx is focusing on innovative technologies for gene therapy, including riboswitch gene regulation technology for precise control of gene expression [20]

Core Viewpoint - Sarepta Therapeutics reported a significant adjusted loss in Q1 2025, primarily due to a one-time charge related to a multi-billion-dollar deal with Arrowhead Pharmaceuticals, missing earnings expectations by a wide margin [1][2] Financial Performance - The company recorded total revenues of $744.9 million, an 80% increase year over year, driven by sales of Elevidys, surpassing the Zacks Consensus Estimate of $698.2 million [3] - Product revenues rose 70% year over year to $611.5 million, although this figure fell short of the Zacks Consensus Estimate of $664 million [4] - Elevidys sales generated $375 million, down from $384.2 million in the previous quarter, missing estimates due to safety concerns and site-related constraints [5] - Collaboration and other revenues associated with Elevidys supply to Roche amounted to approximately $133.3 million, significantly up from $54 million in the year-ago period [6] Operating Costs - Adjusted R&D expenses surged to $749.2 million from $178.1 million year over year, reflecting increased costs related to the Arrowhead deal [8] - Adjusted SG&A expenses totaled $107.1 million, a 7% increase year over year, driven by higher professional service expenses [9] Guidance and Market Reaction - Sarepta revised its full-year net product revenue guidance to between $2.3 billion and $2.6 billion, down from a previous forecast of $2.9-$3.1 billion, citing safety concerns and administrative complexities [10] - Following the revised guidance, shares of Sarepta fell 21% in after-hours trading, with a year-to-date decline of 62% compared to a 3% decline in the industry [11] Pipeline Developments - The company is advancing several investigational gene therapies, including SRP-9003 for Limb-girdle muscular dystrophy and SRP-6004 for LGMD type 2B/R2, with data expected in 2025 [17][18] - Sarepta's licensing agreement with Roche for Elevidys allows Roche exclusive rights to market the product in non-U.S. markets, which has implications for revenue generation [7]

Core Viewpoint - Vertex Pharmaceuticals reported weak first-quarter results, missing estimates for both earnings and sales, with total revenues of $2.77 billion, a 3% year-over-year increase driven by Trikafta/Kaftrio sales and early contributions from Alyftrekt [1][2] Revenue Performance - Trikafta sales increased by 9% in the U.S., while international sales decreased by 5% due to an illegal copy in Russia, impacting intellectual property rights [2] - Vertex raised the low end of its total revenue guidance by $100 million, now expecting between $11.85 billion and $12 billion [2] New Product Performance - Investor focus during the earnings call was on the performance of newer drugs, including Alyftrek and Journavx, which fell short of expectations [3][5] - Alyftrek generated $53.9 million in its first quarter but saw slower-than-expected patient switching from Trikafta [11] - Journavx, launched in mid-March, contributed insignificantly to total revenues, with expectations for sales to pick up in the second half of the year [12][13] Pipeline Development - Vertex is advancing a diverse late-stage pipeline with four pivotal programs, including povetacicept, which is considered to have significant potential [16] - Three phase III programs are on track for enrollment completion this year, setting the stage for potential regulatory filings next year [17] - The company is temporarily pausing the phase I/II study of VX-522 due to tolerability issues [18]

Core Viewpoint - Vertex Pharmaceuticals reported mixed financial results for Q1 2025, with adjusted earnings per share of $4.06, missing estimates, and a year-over-year decline in earnings of 14.7% [1][2] Financial Performance - Total revenues for Q1 2025 were $2.77 billion, slightly below the consensus estimate of $2.82 billion, but up 3% year-over-year, primarily driven by Trikafta/Kaftrio sales [2][4] - U.S. revenues increased by 9% year-over-year to $1.66 billion, while international revenues decreased by 5% to $1.11 billion, impacted by illegal product availability in Russia [4] - Trikafta sales reached $2.54 billion, a 2.4% increase year-over-year, but fell short of estimates [5] - Alyftrek generated $53.9 million in sales, showing steady uptake since its approval [6] - Other product revenues decreased by 17.4% year-over-year to $170.8 million [6] Cost Structure - Adjusted R&D expenses rose by 31.2% year-over-year to $879 million, while SG&A expenses increased by 22.4% to $333 million due to higher investments in clinical studies and commercial launches [10] - Adjusted operating income was approximately $1.18 billion, reflecting a nearly 12% year-over-year decrease [11] Guidance and Outlook - Vertex raised its total revenue guidance for 2025 to a range of $11.85-$12 billion, reflecting growth in CF medicines and new product launches [12] - Combined adjusted R&D, AIPR&D, and SG&A expenses for 2025 are expected to be between $4.9-$5 billion [13] Pipeline Developments - Vertex is advancing its pipeline in various disease areas, including treatments for diabetic peripheral neuropathy and type I diabetes [14][18] - The company is also developing povetacicept for autoimmune diseases and has ongoing studies for its next-gen Nav1.8 inhibitor [16][15] Market Performance - Vertex shares have increased by 24.3% year-to-date, contrasting with a 2.2% decline in the broader industry [3]

Core Insights - Beacon Therapeutics announced promising 6-month interim safety and efficacy results from the Phase 2 DAWN trial of laru-zova for patients with X-linked retinitis pigmentosa (XLRP) [1][2] - Laru-zova demonstrated improvements in visual function, including low luminance visual acuity and microperimetry, and was generally well-tolerated by participants [1][5] Company Overview - Beacon Therapeutics is a clinical-stage biotechnology company focused on developing treatments for blinding retinal diseases, with laru-zova as its lead program [11] - The company has a scientific foundation that includes a late-stage clinical candidate for XLRP and two preclinical programs targeting dry age-related macular degeneration and inherited cone-rod dystrophy [11] Product Details - Laru-zova (laruparetigene zovaparvovec) is a gene therapy designed to restore the function of rods and cones in XLRP by delivering a functional copy of the RPGR gene [2][6] - The therapy has received several designations from regulatory agencies, including Regenerative Medicine Advanced Therapy (RMAT) and Fast Track from the FDA, and Orphan Drug Designation from both the FDA and EMA [6] Clinical Trials - The DAWN trial is an open-label study assessing the efficacy, safety, and tolerability of laru-zova in participants with XLRP who have previously received gene therapy [3][8] - The VISTA trial is a pivotal Phase 2/3 study currently enrolling participants to evaluate the efficacy and safety of laru-zova compared to an untreated control group [9] Disease Context - XLRP is an inherited retinal disease affecting approximately 1 in 25,000 males, leading to progressive vision loss and eventual blindness, with no approved treatments available [10]

VYJUVEK Commercial Performance and Expansion - VYJUVEK has generated over $429 million in revenue since its launch[15] - Q1 2025 net revenue reached $88.2 million, a 95% increase compared to Q1 2024[16, 18] - The gross margin for Q1 2025 was 94%, with a gross to net of 17%[18] - Over 540 reimbursement approvals have been secured as of April 2025, with 45% from commercial payers and 39% from Medicaid[24, 25] - 97% of covered lives under commercial and Medicaid plans have positive access[25] Clinical Development Programs - Four clinical readouts are expected in 2025 for KB407 (cystic fibrosis), KB408 (AATD lung disease), KB304 (aesthetic skin conditions), and KB803 (ocular DEB)[19] - A Phase 1/2 study evaluating KB801 for neurotrophic keratitis is underway, with the first patient expected to be dosed this month[43] - Initial KB408 molecular data showed 35% AAT positive cells at baseline increasing to 39% after KB408 in patient 07[37] Financial Stability - The company's cash and investments totaled $7653 million as of March 31, 2025[54] - Non-GAAP R&D and SG&A expense guidance for the full year 2025 is $150 million to $175 million[55]

Core Insights - Abeona Therapeutics Inc. received FDA approval for Zevaskyn, the first autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [1][3] - Zevaskyn is a single-application treatment that utilizes genetically modified skin cells to produce functional Type VII collagen, allowing for significant coverage of wounds [2][3] - The list price for Zevaskyn is set at $3.1 million per treatment, significantly higher than previous estimates, with projected sales of over $30 million by the end of 2025 [5][6] Market Potential - HC Wainwright estimates peak U.S. sales for Zevaskyn could reach around $600 million, with a potential market opportunity exceeding $2 billion if all 750 identified patients are treated [6] - The updated revenue forecast for Abeona is $31.6 million in 2025, increasing to $130.4 million in 2026 [5][6] Competitive Landscape - Zevaskyn may be used in conjunction with other treatments, such as Krystal Biotech's Vyjuvek, which was recently approved in Europe for similar indications [3][4] - Analyst Raghuram Selvaraju maintains a Buy rating for Abeona, raising the price target from $15 to $20 per share, reflecting increased confidence in Zevaskyn's market position [6]

Company Overview - REGENXBIO Inc. is a biotechnology company focused on gene therapy with a mission to improve lives through its curative potential [3] - The company has been a pioneer in AAV gene therapy since its founding in 2009 [3] - REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for Duchenne, RGX-121 for MPS II, and RGX-111 for MPS I, in partnership with Nippon Shinyaku [3] - The company is also collaborating with AbbVie on surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy [3] - Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA® [3] Upcoming Events - REGENXBIO will host a conference call on May 12, 2025, at 4:30 p.m. ET to discuss its financial results for Q1 2025 and recent operational highlights [1] - Listeners can register for the webcast and analysts can participate in the Q&A session through provided links [2] - A replay of the webcast will be available on the company's investor website approximately two hours after the call [2]